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A randomized placebo-controlled trial comparing cyclosporine plus steroids with or without Myfortic as primary treatment for extensive chronic graft-versus host disease

Approved WMORecruitment stopped

To improve response rate of primary treatment of extensive chronic GvHD with the addition of MPA to the reference treatment CsA+PDN (comparator)Primary ObjectiveTo improve remission rates with cyclosporine A+ prednisone + Myfortic® (CsA+PDN+MPA)…

A phase II, open-label, non-controlled, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurrent aplastic anemia (CETB115E2201)

Approved WMOWill not start

To characterize the PK of eltrombopag at steady state after oral administration in pediatric patients with SAA.Secondary (key only, see protocol page 40-41 for all objectives): Safety and tolerability. Efficacy (overall response rate ORR).